{"id":"W4416431787","doi":"10.1016/j.mam.2025.101424","title":"Precision gene therapy: Tailoring rAAV-mediated gene therapies for inherited retinal dystrophies (IRDs)","year":2025,"lang":"en","type":"review","venue":"Molecular Aspects of Medicine","topic":"Retinal Development and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":1,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"Novartis Stiftung für Medizinisch-Biologische Forschung; Foundation Fighting Blindness","keywords":"Genetic enhancement; Retinal pigment epithelium; Gene delivery; Retinal; Gene; Retina; Genome editing; Transgene; Retinal ganglion cell","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"codex-gemma-dda1882f352a","candidate_categories":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.0005269229,0.0006897872,0.001646428,0.0003963402,0.0000945732,0.00001943197,0.0006032986,0.0005089648,0.00003661751],"category_scores_gemma":[0.0007536396,0.0005202151,0.0005172033,0.0005031541,0.0002968898,0.000004812319,0.0001750284,0.0002247304,0.000003269879],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00003460865,"about_ca_system_score_gemma":0.0004406979,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00001426178,"about_ca_topic_score_gemma":0.000002767418,"domain_scores_codex":[0.9971513,0.0002043115,0.0009526789,0.000797604,0.0004568708,0.0004373068],"domain_scores_gemma":[0.9982051,0.0001322054,0.0005513654,0.0006845527,0.0003122344,0.0001145788],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.0007219425,0.0001765334,0.00001161977,0.01020021,0.001806182,0.00008461539,0.0001296157,0.000005109481,0.2297712,0.0003263987,0.003602731,0.7531639],"study_design_scores_gemma":[0.002703549,0.001381201,0.0000222964,0.00699004,0.0005014245,0.00003956899,0.00005837261,0.000003135927,0.02925617,0.0004153592,0.9579706,0.0006583175],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.001530413,0.9915648,0.003277082,0.0002731027,0.0003830841,0.001659468,0.00005854491,0.00003337138,0.001220154],"genre_scores_gemma":[0.001246299,0.9932578,0.001679916,0.0001533963,0.0003377846,0.0002656166,0.002445458,0.00009966236,0.0005140785],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.9543678,"threshold_uncertainty_score":0.9997249,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02051490841312063,"score_gpt":0.3027497237540689,"score_spread":0.2822348153409482,"validation_status":"score_only:v0-immature-baseline","note":"Baseline scores from an immature model (maturity gate not passed). Scores rank; they never assert a category."}}